🧬 This Week in Rare Diseases: Breakthroughs, Designations & Policy Shifts!
This week’s roundup delivers crucial insights into the future of orphan drugs and clinical innovation. Discover how the FDA, biotech leaders, and policymakers are shaping treatments for conditions with limited options.
🧪 In This Episode
💊 FDA grants Orphan Drug Designation to Veverimer for anti-GBM disease
🧠 Chiesi expands CNS drug development with Key2Brain collaboration
💥 Vascarta’s non-opioid curcumin gel gains Orphan Status for sickle cell disease
🧬 Capricor makes strides in Duchenne and Becker muscular dystrophy programs
🦴 Promising Phase 1 data for Angitia’s osteogenesis imperfecta therapy
🌬️ Circle Pharma enters small cell lung cancer space with CID-078
⚖️ Senate drops Orphan Cures Act from IRA Reform — biotech backlash vs. patient praise
🩸 Novartis’ Fabhalta shows strong Phase IIIB results in PNH, including a 2.01 g/dL hemoglobin boost with no transfusions
Whether you’re a clinician, researcher, or innovator in the rare disease space, this update gives you a front-row seat to the most pivotal developments.
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